When the first gene therapy using CRISPR was approved in the US in 2023, former President Joe Biden struck an optimistic tone.
“This holds tremendous promise for developing additional life-saving treatments…for millions of Americans who live with other rare diseases,” he said at the time.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
Fast forward to 2025, and the sentiment surrounding cell and gene therapy has shifted towards more grounded realism.
Back and forth between Sarepta and FDA
This year, headlines in the cell and gene therapy landscape have so far been dominated by Sarepta. The biotech’s Duchenne muscular dystrophy treatment Elevidys (delandistrogene moxeparvovec) became the centre of a regulatory storm following alleged links to patient deaths.
“It’s a tough job being a regulator, and it’s a tough job being a pioneer in this space. You are doing a lot of learning in the public domain about a platform which clearly has huge potential, and the regulator that wants to get drugs to patients as quickly as they can, and there is a degree of a judgment call,” says Owen Smith, partner at 4BIO Capital.
The safety of Sarepta’s therapy was eventually cleared by the US Food and Drug Administration (FDA) after an investigation. Still, the very public back and forth between the agency and Sarepta fuelled previous concerns about the volatility in the cell and gene space, given the unique technology that underpins it.
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalDataThere has also been a change at the FDA’s Center for Biologics Evaluation and Research (CBER), which is responsible for cell and gene therapy regulation in the US. CBER head Vinay Prasad left the FDA amid the Sarepta controversy, only to be reinstated just two weeks later.
Shares in cell and gene therapy companies have oscillated against the backdrop of regulatory instability, with financial analysts, such as those from William Blair, forecasting volatile times for the industry. These models take into account regulatory uncertainty in the US, along with the well-treaded issues of manufacturing and scalability in cell and gene therapy development.
Learning from previous failures
Amid scalability hurdles and commercial hesitancy, some companies have turned away from cell and gene therapy development.
Vor Bio, for example, has pivoted to the lucrative autoimmune sector, calling it a day on its advanced therapy pipeline. The company was one of the many casualties of the sector’s poor performance, eventually even winding down clinical and manufacturing operations in May, along with firing 95% of its staff. A $4bn licensing deal for an autoimmune drug developed by a Chinese biotech has offered shareholders a lifeline, however.
The largest company to arguably fall by the cell and gene therapy wayside is US biotech bluebird bio. The company was riding high on a regulatory wave following approval of its sickle cell gene therapy Lyfgenia (lovo-cel) in December 2023, though it has since struggled to turn a profit. High development costs and slower-than-expected market adoption for its products proved terminal for the company, which was eventually sold for a pittance in February 2025.
Stella Vnook, CEO of cell and gene logistics company Likarda, says: “Cell and gene therapy was all over the news [during sickle cell approvals]. It was exciting. The science was brilliant.
“The hope was that cell and gene therapies would advance, but questions started being asked about reimbursement and logistics. That’s why a lot of these companies had to either pivot, consolidate or abandon some of those programmes.”
Matthew Durdy, CEO for the Cell and Gene Therapy Catapult, an organisation tasked with advancing the modality’s growth in the UK, argues that more therapeutics reaching market is the fix, but that only happens if companies have enough economic ceiling in R&D and manufacturing to reduce prices.
Durdy says: “The profitability of the therapeutics for the industry is low because of the high cost of manufacturing. The ideal solution to that problem is lots and lots of therapeutics being developed in the space. And as you start to see more therapeutics for each disease indication, then the buyers procure products on the basis of value for money. But the cost of goods has to come down.”
Vnook concurs, explaining that: “We’ve made a lot of scientific progress, but we haven’t advanced with enough scalability equipment to resolve some of the inherent challenges. This even includes logistics – for example, how to ship cells overseas.”
Lack of investment or low interest?
Equipped with its unique delivery platform, US biotech Medera looked set for public trading this time last year. In September 2024, the biotech announced a $623m deal with special purpose acquisition company (SPAC) Keen Vision. There have been no updates on the deal since.
Medera’s CEO Ronald Li confirmed to Pharmaceutical Technology Focus that the ambition to go public is still there, though the company is waiting for the right market window.
Li said: “Obviously, the market has been quiet, and that’s for a range of different reasons. We are still positioned for a listing in the foreseeable future. Exact timing will depend on a number of factors and we’re waiting for the market to open up.”
This reflects an issue in sectors with such innovative technologies. One of the reasons for the lack of public listings in the cell and gene therapy space is that in a space with such untreaded technologies, the bar to convince investors – and regulatory agencies down the road – is much higher.
“We still believe there is demand for credible, late-stage cell and gene therapy companies with scalable platforms. The bar is getting higher, but at the same time, people appear to understand better what they’re looking for as well,” Li says.
Wider investment trends in the pharmaceutical industry also need to be considered. Cell and gene therapies are just one modality that is suffering from dry funding channels, with cancer vaccines, rare diseases, and women’s health all lacking proper investment in recent years.
“There’s a bit of a choke up in terms of investment into the cell and gene therapy space. But that’s not cell and gene therapy specific. That’s across the whole of the biotech sector,” says Durdy.
Showcasing that investment oppurtunities still exist, however, Smith says that 4BIO Capital has targeted gene therapy companies more recently compared to previous funds during a time when the sector could have been tagged as overvalued.
“Fast forward to our Fund III, we’ve made a number of gene therapy bets because we can see that there is a valuation arbitrage,” he says.
Cause for optimism
Despite the backdrop of instability at US regulators and some companies leaving the space, experts agree that the science of cell and gene therapies has come on leaps and bounds in the past decade.
“I’d say we’re pretty much bang on track,” says Durdy. “If you look at the progress, the underlying progress the industry has gone through, it’s actually incredibly good.”
The first gene therapy approved by the US Food and Drug Administration (FDA) was Kymriah (tisagenlecleucel) for certain types of leukaemia in 2017. The high-profile sickle cell therapy approvals then brought public attention in 2023. As of August 2025, there are 45 FDA-approved cell and gene therapies in the US, with seven being greenlit in 2024 alone.
“The field is moving forward, positively, meaning that investors, investigators, and others involved are all starting to understand what they are looking for,” says Li.
Correction rather than collapse
While headlines have often spotlighted bluebird bio’s stuttering end, it is by no means a reflection of what lies in store for other cell and gene therapy specialist companies.
Regulatory bodies are becoming ever more welcome to the modality, while manufacturing technologies continue to advance, even if not at the same pace as the science. Governments also acknowledge the potential of cell and gene therapies to reduce healthcare burden.
“I don’t think a bubble has burst, but I think it’s clearly deflated,” says Owen Smith. “I think we’re in a correction phase at the moment, rather than the space being collapsed.”
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
